The present invention provides compositions comprising mRNA molecules encapsulated within lipid particles. The lipid particles comprise a cationic lipid, a non-cationic lipid, and an mRNA molecule that is encapsulated within the lipid particle. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease. The invention also provides cationic lipids that are useful for preparing the compositions of the invention.
本发明提供了包含封装在脂质颗粒内的 mRNA 分子的组合物。脂质颗粒包括阳离子脂质、非阳离子脂质和封装在脂质颗粒内的 mRNA 分子。这些组合物可用于将 mRNA 分子导入人体,在人体中翻译产生
多肽,从而改善疾病的一种或多种症状。本发明还提供了可用于制备本发明组合物的阳离子脂质。